An expensive new drug is finally available for her rare disease - but she's been battling it for two years

When she was only six months old, Annie Wilson was diagnosed with spinal cord atrophy, a rare condition that causes progressive muscle weakness and movement disorders – and she said she would not be more than three years old. More than 30 years later, the first therapy took place. Your condition was approved by Biogen Inc.

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and Ionis Pharmaceuticals Inc.

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Spinraza. Spinraza has become aware of innovation, but also the high price: up to $ 750,000 in the first year and $ 375,000 per year. At the time of approval, Wilson had trouble breathing, talking and even driving the wheelchair and was excited about the prospect of the new treatment. After initially telling her she was a good candidate, her doctor, who works for the California-based health system Kaiser Permanente, said she was too weak to come back later, Wilson told MarketWatch. Wilson has challenged Kaiser's decision through a Medicare appeal. Even so, the two-year battle was exhausting, Wilson said in an e-mail. She suspects that the stress is affecting her health and has even considered giving up. "I did not want to die to fight for a drug that stops the progression of my disease," Wilson said. "I have a feeling that denying someone is like telling Kaiser that your life does not matter to us, but the dollar is." "If I got Spinraza, I expect no miracle," she said. "To stop the progression of my illness is what I aspire to. The preservation of the little movement that I have left is of the utmost importance to my quality of life. "

Annie Wilson, 36, of Alameda, California, first diagnosed the spinal cord muscles at the age of six months with the rare disease. She has been battling the Kaiser Permanente health care system over the past two years to gain access to an expensive therapy that could stop the disease from progressing. More: Extremely expensive drug against rare diseases could "invite a storm of criticism". Kaiser has "tremendous empathy" for Wilson and wants her to receive support and care, but "we can not recommend a treatment that puts a patient at risk and has no clinical trials to prove its efficacy to the patient," said Dr. Sameer Awsare, vice president Managing Director of Permanente Medical Group, "There are currently no clinical studies showing that Spinraza is effective in ventilator-dependent adults with spinal muscular atrophy, and in some cases there are significant, potentially fatal risks." In recent years, more and more drugs But Wilson's experience speaks to the jumble of complications that can keep promising, but expensive, therapies out of their patients Related topics: Why health insurers will not cover this rare disease drug worth $ 300,000 a year It is presumed n that about 10,000 to 25,000 people in the US have SMA, a congenital disorder, according to the SMA Foundation. Biogen estimates that the majority of the total population or about 60% are adults. Read: This expensive drug is now the most watched product by Biogen – and could soon become a blockbuster. Spinraza was approved in late 2016 for both children and adults. Some health insurers – including emperors – were bothered to cover adults because clinical trials approved to receive Spinraza have been tested only in children. More adults with SMA are now being treated with Spinraza, including Wilson's patient advocate Kaiser, Joahn Ginsberg said. "They need to treat her, and they have to pay for this treatment because she pays every month in Medicare," Ginsberg said. However, the policies differ, and large health insurers like UnitedHealthcare

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and Cigna Corp.

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have guidelines that do not apply to people who require permanent use of respirators. In particular, price is a key factor in the limitations of spinraza access, perhaps because in a rare disease SMA is a relatively common one. See: ALS patient group dissatisfied with the $ 115 million raised by the Ice Bucket Challenge. However, adults with SMA are treated in the US, including those who rely on ventilators. Stanford University Hospital, for example, had treated 32 adults in mid-2018, 22 of whom were unable to walk, and three of whom were ventilator-assisted, with no serious side effects or medical concerns. The neuromuscular team sent Wilson's doctor to Kaiser earlier this year. The Stanford team also recommends the "treatment of [Annie] with Spinraza is reasonable and would help her stop the progression of the disease and possibly improve the existing function, "the letter said. Biogen has attempted to extend the use to adults and estimated that by the end of October, about 15% of adult patients had been reached, compared to about half of all infants and pediatric patients. A spokesman told MarketWatch that Spinraza "has demonstrated significant and clinically meaningful efficacy … in a wide range of SMA populations." said at a teleconference in late October. But questions about price, patient population, and side effects are likely to continue, and for Wilson, time is everything. "Will it miraculously heal her SMA? No, "said Ginsberg. "But do we think it can help her? Yes."